$SGMO status going into C22
Two synthetic gene transfer programs:
- HemA now run by $PFE P3. Hold for protocol amendment. Expect timing update for full analysis in next couple months.
- #Fabry owned program good data potential first/best in class
No further programs.
$SGMO development focus leveraging ZF technology shows evolution from old four platforms to new integrated tech message. ZF tech facilitates both ex vivo and in vivo genomic medicines. Examples are CAR Tregs cell therapy and ZFP-TF CNS regulation.
2/ ImageImageImageImage
Cell therapy pipeline:
-Sickle cell collab with $SNY lead indication (see next)
-$GILD collab programs on hold. No update for a while but likely tied to durability issues seen at $ALLO $CRSP $DTIL
-Steadfast study is #POC for CAR Tregs with significant pipeline to follow
3/ ImageImageImageImage
$SNY concluded that their preliminary POC efficacy and safety results confirm the potential tx value. Appears headed for registrational study in next 12-18 months. $SGMO
4/ ImageImageImageImage
Reminder to look at previous tweet figure 6 showing number of #SCD vaso-occlusive crises (VOC) dropped from 44 to 1.
5/ Image
ZFP-TF initial in vivo. Partners progressing but appear delivery hurdle remains
$TAK -686 clinical trial being planned for huntington’s
ALS subject of breakthrough article on $PFE website
$NVS 2 VPs joined Sangamo
$BIIB working on 4 selected programs. Waiting for updates
6/ ImageImageImageImage
Gene regulation pipeline:
- Owned #Prion and one undisclosed preclinic.
- $BIIB has selected four programs (2 preclinic) w/ 8 optional targets which $SGMO likely working on
- $NVS has three active programs
- $PFE ALS ZFP-TF selected and in animal testing
- $TAK see prior
7/ Image
Key for all bios in this kind of market is cash runway IMO. $SGMO relies on collaboration partners to fund opex thru reimbursed research and #milestones. Impact of HemA hold on forecast is key. No change yet to prior fcst: will update once we get more clarity.
8/ ImageImage
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More from @mchilberg

12 Apr
The chase for an #HIV cure has been elusive for decades with as many failures as seen with CNS diseases. $SGMO has been dedicated to this effort tho are now supporting sponsors given the cost of the effort. This stream will provide an recap. #Autologous #CellTherapy
$SGMO began this clinical effort in 2009. They have worked with UPenn, City of Hope, Case Western University. NAID and CIRM have helped with funding among others. We should see some critical updates this year.
2/ Image
The first trial (sponsored by UPenn) SB-728 began in 2009 focused on HAART treated patients and was focused on safety and comparison/persistence of CD4/CD8 T cells after ZFN modifed autologous T cells were infused.
#HIV #CellTherapy
3/ Image
Read 13 tweets
16 Mar
$BEAM 4Q20 results out. Valuation is crazy but they are the best of #Crispr breed in my opinion given execution to date.
1/ ImageImage
$BEAM focus on platform, delivery and manufacturing is enabled by share price and ability to source funds without huge dilution
2/ ImageImageImage
$BEAM focus on based editing of transition mutations.
#dreaminase ImageImageImage
Read 6 tweets
15 Mar
$SNY looks like they are ready for a Genomic Medicine Unity #GMU coming out year in C21. Many hires. Many open reqs. Building on Ablynx, Bioverativ and Kiadis M&A
$SNY #GMU head of Rare and Neurologic Disease Research is Christian Mueller. Video into is pretty good
Addl heads include Bruno Figueroa (CMC) and Cate O'Riordan (translational unit)

$SNY #GMU Principal Scientist Robert Jackson profile says he is working on: Huntingtin's, MLD, PKU, Neurophathies, T1Diabetes Hypertension, Nephrophaty, vascular disease, Rheumatoid Arthritis and sensorineural hearing loss
Read 14 tweets
25 Feb
Part II of the $CRSP $SGMO comparison will hopefully be a bit less dry now that Part I updates are in place. This thread will focus more on valuation, outlook and why the valuation cycle should matter to investors.
Let's start with the concept of #Disruptive technologies. The originator of disruptive innovation theory, Clayton Christensen worked with HBR in 2015 to revisit the past 20 years.
#GenomicMedicine is disrupting Big Pharma who has begun to respond by spinning off old product lines and jumping into #GeneTherapy.
Read 24 tweets
25 Feb
This is the third edition of my tweetstorms on #GeneTherapy #CellTherapy #GeneEditing. This one compares $SGMO to $CRSP and attempts to look at #GenomeMedicine from the perspectives laid out by C.Woods at ARK This is part one of the series. Pt 2 tomorrow AM 1/20
The charts of both are attached and useful context. $CRSP went public in 2016. The weekly chart is attached and notes the pct ownership of $ARKG $ARKK since CY18. Prior the stock was stagnant below the support/resistance levels show. 2/20
I'm including two charts for $SGMO. Wkly and Mthly. The weekly shows they were pulled along with the #geneediting group The monthly shows this rally/bust cycle is not unique. Compare the SGMO 2017-2018 to CRSP. Pretty similar. More later. 3/20
Read 21 tweets
20 Feb
Appears Cowen published a useful courtesy $SGMO rept for investors prior to their annual HC conference. Most of the verbiage is an accumulation of prior reports. They have higher model royalties for $PFE and $SNY royalties than mine. Not a SOTP or full blown model Image
Cowen states that though the prelim data on $SNY collab was unimpressive, they are cautiously optimistic. My fcst was pushed out several years consistent with $BLUE issues and probability that more issues will crop up for $CRSP and $SGMO programs.
Attached screenshot of Cowen P&L is consistent with reported $SGMO revenue in C19 including:
- Research reimbursement $16m (mostly Gilead/Sanofi)
- Upfront amort $46m
- Milestones $39m Image
Read 4 tweets

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